发明授权
- 专利标题: Modified iRNA agents
- 专利标题(中): 修饰的iRNA试剂
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申请号: US12510050申请日: 2009-07-27
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公开(公告)号: US08017762B2公开(公告)日: 2011-09-13
- 发明人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Meena
- 申请人: Muthiah Manoharan , Kallanthottathil G. Rajeev , Meena
- 申请人地址: US MA Cambridge
- 专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人地址: US MA Cambridge
- 代理机构: LeClairRyan
- 代理商 Jeffrey N. Townes
- 主分类号: C07H21/04
- IPC分类号: C07H21/04 ; A61K31/70
摘要:
The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.The inclusion of such a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated, e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety. e.g., cholesterol, is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents.
公开/授权文献
- US20100076056A1 MODIFIED iRNA AGENTS 公开/授权日:2010-03-25
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